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1 . Academic Journal
Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial
저자
by
Melissa P. Wasserstein
;
Robin Lachmann
;
Carla Hollak
;
Antonio Barbato
;
Renata C. Gallagher
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)
Open Access (BioMed Central)
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7 . Academic Journal
Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults
저자
by
Robin H. Lachmann
;
George A. Diaz
;
Melissa P. Wasserstein
;
Nicole M. Armstrong
;
Abhimanyu Yarramaneni
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-11 (2023)
Open Access (BioMed Central)
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7 . Academic Journal
Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
저자
by
George A. Diaz
;
Roberto Giugliani
;
Nathalie Guffon
;
Simon A. Jones
;
Eugen Mengel
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-11 (2022)
Open Access (BioMed Central)
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7 . Academic Journal
P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials
저자
by
Melissa Wasserstein
;
Roberto Giugliani
;
Simon Jones
;
Robin Lachmann
;
Maurizio Scarpa
, et al.
소스
Genetics in Medicine Open, Vol 1, Iss 1, Pp 100377- (2023)
Full Text (Clinical Key)
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7 . Academic Journal
Correction : Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
저자
by
George A. Diaz
;
Roberto Giugliani
;
Nathalie Gufon
;
Simon A. Jones
;
Eugen Mengel
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-2 (2023)
Open Access (BioMed Central)
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7 .
A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results
저자
by
Melissa Wasserstein
;
Robin Lachmann
;
Carla Hollak
;
Laila Arash-Kaps
;
Antonio Barbato
, et al.
소스
Genetics in medicine, 24(7), 1425-1436. Lippincott Williams and Wilkins
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7 .
Pediatric and adult acid sphingomyelinase deficiency (ASMD) patients exhibit mechanistic similarity in their disease and response to olipudase alfa
저자
by
Mengdi Tao
;
Randolph J. Leiser
;
Chanchala Kaddi
;
Bradley Niesner
;
Jing Li
, et al.
소스
Molecular Genetics and Metabolism
. 138:107334
Web of Science
Scopus
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7 .
Safety and clinical outcomes in children with acid sphingomyelinase deficiency were not impacted by a change in the olipudase alfa drug manufacturing process: ASCEND-Peds 1-year subanalysis
저자
by
Monica Kumar
;
Nicole Armstrong
;
Yong Kim
;
Jing Li
;
Susan Richards
, et al.
소스
Molecular Genetics and Metabolism
. 138:107188
Web of Science
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7 .
Plasma lyso-sphingomyelin as a biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials
저자
by
Melissa P. Wasserstein
;
Roberto Giugliani
;
Simon Jones
;
Robin Lachmann
;
Maurizio Scarpa
, et al.
소스
Molecular Genetics and Metabolism
. 138:107355
Web of Science
Scopus
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7 .
Quantitative systems pharmacology (QSP) analysis predicts that olipudase alfa treatment of patients with extreme acid sphingomyelinase deficiencies can result in meaningful debulking of visceral tissue sphingomyelin
저자
by
Randolph J. Leiser
;
Mengdi Tao
;
Chanchala Kaddi
;
Jing Li
;
Catherine Ortemann-Renon
, et al.
소스
Molecular Genetics and Metabolism
. 138:107198
Web of Science
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